National Institute For Health And Care Excellence 's Decisions Research Articles

OP137 A Qualitative Exploration Of The National Institute For Health And Care Excellence’s Impact On International Health Technology Assessment

IntroductionThe National Institute For Health And Care Excellence (NICE) is widely acknowledged as a seminal health technology assessment (HTA) body, known for its transparent and accountable approach to decision-making. This research aimed to investigate the impact of NICE methodology and decisions on international HTA bodies. We sought to identify direct and indirect factors that may influence an international HTA body’s methods or outcomes. To the best of our knowledge, this is the first research to use a qualitative approach to understand the influence of NICE on other HTA bodies.MethodsWe conducted 13 semi-structured qualitative interviews with HTA and market access experts from industry and academia from nine countries (Brazil, Israel, Italy, Japan, Poland, Saudi Arabia, South Korea, Sweden, and the United Arab Emirates). The interview script was organized into three main sections: comparing NICE methods and processes with other HTA bodies; the impact of specific NICE decisions; and Likert scale questions (to allow for comparability of opinions).ResultsMost interviewees believed their local HTA body would consider NICE’s decision when evaluating a medicine. However, the way and extent to which NICE influences HTA varied across countries. The most common means of considering a NICE decision was as background information or context for an HTA evaluation. Generally, interviewees suggested that negative NICE decisions had more impact on local decision-making than positive decisions. Nine of the 13 interviewees agreed or strongly agreed that their country’s HTA body considers the decisions of other HTA bodies in their decision-making process. Eleven of the 13 interviewees agreed or strongly agreed that the development of their country’s HTA body methods and processes was influenced by NICE.ConclusionsNICE is perceived to be a seminal HTA body, with continued influence on HTA agencies in other countries. However, the mechanisms and extent of this influence varies considerably between countries. We suggest that implicit factors are likely to contribute more to NICE’s influence than individual decisions. Nevertheless, further research is needed to reveal these factors and increase efficiency in international HTA decision-making processes.

Evaluation of US oncology electronic health record real-world data to reduce uncertainty in health technology appraisals: a retrospective cohort study

ObjectivesExamine whether data from early access to medicines in the USA can be used to inform National Institute for Health and Care Excellence (NICE) health technology assessments (HTA) in oncology.DesignRetrospective...

‘… they had interpreted “disability” as referring to a patently visible disability’: experience of a patient group with NICE

Erythropoietic protoporphyria (EPP) is an ultra-rare genetic disorder characterised by intolerance to visible light. Starting in early childhood, people with EPP suffer from social isolation, impaired educational and occupational opportunities, and low quality of life. Afamelanotide is the only effective and approved therapy for EPP. In England, its cost-effectiveness is currently assessed by the National Institute for Health and Care Excellence (NICE), which in 2018 issued a negative recommendation for funding. Stakeholder organisations, including our patient organisation, submitted appeals against the recommendation, which were upheld in all possible grounds. Moreover, the appeal panel expressed concerns about whether the evaluating committee discriminated against people with EPP and suggested that it seek guidance regarding the Equality Act 2010. However, three years later, the identified issues have not been addressed and patients in England remain without treatment. Afamelanotide represents another example for the trend towards a loss of fairness in NICE decisions.

The normative grounds for NICE decision-making: a narrative cross-disciplinary review of empirical studies.

The National Institute for Health and Care Excellence (NICE) is the UK's primary health care priority-setter, responsible for advising the National Health Service on its adoption of health technologies. The normative basis for NICE's advice has long been the subject of public and academic interest, but the existing literature does not include any comprehensive summary of the factors observed to have substantively shaped NICE's recommendations. The current review addresses this gap by bringing together 29 studies that have explored NICE decision-making from different disciplinary perspectives, using a range of quantitative and qualitative methods. It finds that although cost-effectiveness has historically played a central role in NICE decision-making, 10 other factors (uncertainty, budget impact, clinical need, innovation, rarity, age, cause of disease, wider societal impacts, stakeholder influence and process factors) are also demonstrably influential and interact with one another in ways that are not well understood. The review also highlights an over-representation in the literature of appraisals conducted prior to 2009, according to methods that have since been superseded. It suggests that this may present a misleading view of the importance of allocative efficiency to NICE's current approach and illustrates the need for further up-to-date research into the normative grounds for NICE's decisions.

Screening for early onset neonatal sepsis: NICE guidance-based practice versus projected application of the Kaiser Permanente sepsis risk calculator in the UK population

ObjectiveTo compare management recommendations of the National Institute for Health and Care Excellence (NICE) guidelines with the Kaiser Permanente sepsis risk calculator (SRC) for risk of early onset neonatal sepsis...

After 20 Years of Using Economic Evaluation, Should NICE be Considered a Methods Innovator?

The National Institute for Health and Care Excellence (NICE) is only one of several organisations internationally that uses economic evaluation as part of decision making regarding funding and pricing of new medical technologies. However, it can be argued that NICE has developed a more prominent international profile than most in their use of economics. After 20years of operation, it is timely to assess the extent of NICE's achievements, including the economic evaluation methods it has used and its willingness to adapt these as new evaluative approaches emerge and when NICE faces particular policy challenges. This paper considers some of the important policy and contextual developments in the UK over the last 20years and how these may have shaped NICE's approach to economic evaluation. It then assesses key areas of NICE methods, including perspective, defining benefits, modelling and uncertainty. The paper concludes that NICE has provided important support for the development of new methods, in particular through its role in identifying priorities for methods research funding and its sponsorship of the NICE Decision Support Unit. However, potentially important developments in methods in a number of important areas have yet to be formally included in NICE's methods guidance and this should be addressed in the Institute's 2019/2020 methods review.

Efficacy of Pomalidomide, Bortezomib and Dexamethasone in Relapsed or Refractory Multiple Myeloma Post-Lenalidomide: Results from a Systematic Literature Review and Indirect Treatment Comparison

Efficacy of Pomalidomide, Bortezomib and Dexamethasone in Relapsed or Refractory Multiple Myeloma Post-Lenalidomide: Results from a Systematic Literature Review and Indirect Treatment Comparison

PRO117 ACCESS TO ORPHAN DRUGS IN THE UNITED KINGDOM: A REVIEW OF NICE DECISIONS FROM 2017-2018

This study examined how National Institute for Health and Care Excellence (NICE) decisions and resultant patient access differ by drug characteristics and appraisal process in the UK. Published NICE technology appraisal guidance documents for therapies evaluated from January 2017-December 2018 were reviewed; characteristics and results were assessed in a standardised manner to categorise drugs by orphan status, disease condition (oncology vs non-oncology), review type, and NICE decision (“recommend without restrictions,” “recommend with restrictions,” and “not recommended”). The proportion of each decision type was compared numerically between categories. During 2017-2018, NICE published 191 decisions, 73% for drugs indicated for non-orphan conditions and 27% for orphan conditions. Forty-eight percent of orphan indications were recommended without restriction vs 36% of non-orphan indications. Of the drugs for orphan conditions, 75% were for oncology, and a higher proportion of oncology indications were recommended without restriction (54%) vs non-oncology indications (31%). Of 13 orphan drugs for non-oncology conditions, 62% were for adults; 54% were reviewed through the highly specialised technologies (HST) process and the remainder through single technology appraisal (STA). HST utilises a higher threshold for incremental cost-effectiveness ratio to evaluate medicines compared with STA. Of the 6 orphan drugs for non-oncology conditions reviewed through STA, none were recommended without restrictions, 5 were recommended with restrictions on their marketing authorisation and contingent upon the availability of a patient access scheme, and 1 was not recommended. Conversely, 4 of 7 orphan drugs for non-oncology conditions reviewed through HST were recommended without restrictions, and 3 were recommended with restrictions. All orphan drugs for non-oncology conditions in paediatrics (38%) were evaluated through HST. These findings suggest that orphan non-oncology medicines evaluated through STA have a lower likelihood of achieving a recommendation within the full marketing authorisation than orphan oncology medicines or those evaluated through HST.

PBI78 CAR-T TREATMENTS: INSIGHTS FROM INITIAL HEALTH TECHNOLOGY ASSESSMENT DECISIONS IN THE UK, GERMANY AND FRANCE

Two chimeric antigen receptor T-cell (CAR-T) therapies, axicabtagene ciloleucel (axi-cilo) and tisagenlecleucel (tisa-lec), received EU regulatory approval in 2018. Axi-cilo is indicated for relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma in adults, after ≥2 systemic therapies. Tisa-lec is indicated for R/R DLBCL and R/R B-cell acute lymphoblastic leukemia (ALL) in patients aged ≤25 years. Both treatments have received positive reimbursement decisions from the National Institute for Health and Care Excellence (NICE), Federal Joint Committee (G-BA), and French National Authority for Health (HAS). This study assesses these decisions, highlighting the HTA bodies’ insights into these first-generation CAR-T therapies. We reviewed NICE, G-BA, and HAS decisions on axi-cilo and tisa-lec published December 2018–May 2019. NICE recognized axi-cilo and tisa-lec as innovative treatments with clinical effectiveness and recommended them for use within the Cancer Drugs Fund for a limited duration. Additional trial and real-world data are required due to uncertainties in the clinical and cost-effectiveness evidence. Orphan products in Germany automatically have added benefit by definition. G-BA assessed both treatments’ added benefit as non-quantifiable due to insufficiently robust evidence. Funding was approved pending further data collection and assessment not later than 2020 for tisa-lec and 2022 for axi-cilo. HAS assessed axi-cilo as having moderate added clinical value (ASMRIII) in its two indications, tisa-lec as ASMRIII in ALL and having minor added value (ASMRIV) in DLBCL. HAS also cited uncertainties in the evidence and called for yearly reassessments based on real-world data. All three HTA bodies are aligned in recognizing the benefit of these novel therapies despite uncertainties regarding the clinical evidence. They are willing to recommend these treatments for use on an interim basis (until 2022 in the case of axi-cilo in Germany) pending additional data collection and analysis.

PCN222 USE OF NON-NMA INDIRECT TREATMENT COMPARISON METHODOLOGY IN NICE TECHNOLOGY APPRAISALS FOR HAEMATOLOGICAL CANCER

Network meta-analysis (NMA) indirect treatment comparisons (ITCs) employ a network of trials, comparing common interventions in similar patient populations, to assess relative efficacy in the absence of head-to-head trials. Where single-arm trials or heterogeneous trial populations constitute the best-available evidence, individual patient data (IPD) from trials can be used to adjust for baseline characteristics and enable a comparison of treatment efficacy. The acceptance of ITC methods other than NMA by the UK National Institute for Health and Care Excellence (NICE) Evidence Review Groups (ERGs) in technology assessments (TAs) for haematological cancer therapies has not been described. A targeted review of NICE TAs in lymphoma and myeloma published between 2009 and 2019 examined the ITC methods used in manufacturer submissions and subsequent ERG comments on methodological quality. All types of ITC, except NMA, were considered. Of the 25 TAs identified in the review, 5 were excluded due to appraisal termination. Amongst the 20 TAs included, 13 (65%) included ITC methods to synthesise data; eight (40%) matched-adjusted indirect comparisons (MAIC); four (20%) multiple treatment comparisons (Bucher, Bayesian or other); and three (15%) naïve indirect comparisons. Two appraisals presented more than one ITC method. Non-NMA ITC techniques were accepted by ERGs where direct randomised comparative evidence was not available despite substantial uncertainty around treatment effect estimates, and inconsistencies in the choice of covariates and cohorts for matched analyses. Non-NMA ITC techniques are commonly used in submissions to NICE in haematological cancers, with MAIC most frequently used by manufacturers. Matching, or adjusting on baseline characteristics using IPD, can be used to demonstrate the relative effectiveness of new therapies in the absence of evidence from comparative trials. Non-NMA ITC methods preferred by ERGs are not apparent despite published guidance on population-adjusted indirect comparisons by the NICE Decision Support Unit.

PP189 Filling In The Blanks: Is RWE From MAAs Used In NICE Decision Making?

IntroductionThe National Institute for Health and Care Excellence (NICE) may recommend temporary funding through managed access agreements (MAAs) for oncology drugs (via the Cancer Drugs Fund [CDF]) and highly specialized therapies for rare diseases. MAAs allow for the collection of evidence to address key areas of clinical uncertainty, while providing access of medicines to patients, prior to re-appraisal by NICE. Observational data and other real-world evidence (RWE) are crucial requirements for all MAAs and herein we examine the extent these data are being used to inform HTA decisions at re-appraisal.MethodsExisting MAAs entered into between the National Health Service (NHS) England and manufacturers as of 30 October 2018 were identified; for drug:indication pairings with NICE re-appraisals, all information was reviewed and the key data extracted.ResultsOf the twenty-two MAAs identified, only two drug:indication pairings have been subsequently re-appraised by NICE: BV(brentuximab vedotin):non-Hodgkin lymphoma (’recommended’) and pembrolizumab:relapsed or refractory classical Hodgkin lymphoma (’recommended’). Data from a retrospective questionnaire regarding the proportion of patients that received curative stem cell transplant (SCT) post-BV (from patients who received BV in the old CDF) were accepted to provide sufficient evidence on the post-BV SCT rate by NICE. Meanwhile, for pembrolizumab, long-term survival benefit was the key clinical uncertainty; the primary data collection source was updated phase III randomized controlled trial data. At re-appraisal no reference was made to the observational data component; more mature survival data reduced uncertainty over survival benefits and were sufficient to support a positive NICE recommendation.ConclusionsOf the twenty-two MAAs to date, only two drugs have been re-appraised thus far, with both receiving positive NICE recommendations. Observational data were successfully used to address key clinical uncertainties regarding subsequent real-world treatment patterns for BV, but observational data were not referred to in the NICE recommendation for pembrolizumab. The re-appraisal of more drugs in the future will clarify the importance being placed on observational data collection requested by NICE for existing MAAs.

PP81 Real World Data: The Early Access To Medicines Scheme Catches The Worm

IntroductionThe Early Access to Medicines Scheme (EAMS) aims to provide access to medicines prior to market authorization for patients with severe, life-threatening diseases who do not have adequate treatment options. An EAMS designation enables the potential collection of United Kingdom-specific real world evidence (RWE) prior to health technology assessment (HTA) by the National Institute for Health and Care Excellence (NICE). This research evaluates whether RWE is being gathered through the EAMS and utilized to support HTA submissions.MethodsAll EAMS designations as of 7 November 2018 were identified from the Medicines and Healthcare products Regulatory Agency website. For products with final NICE guidance, all publicly-available NICE documentation was reviewed.ResultsSixteen product and indication pairings with an EAMS designation were identified, with 12 having received final NICE guidance (11 were recommended, 3 were recommended for temporary reimbursement via the Cancer Drugs Fund, and 2 were not recommended). Of the 11 recommended products, seven had references to the number of patients or sites with product access through the EAMS, but only one (dupilumab for atopic dermatitis) had detailed data collected during the EAMS period. The manufacturer of dupilumab reported baseline demographics and disease characteristics from a cohort of 35 patients treated under the EAMS to inform the generalizability of trial populations for clinical practice. Follow-up results from this cohort demonstrated that real-world data on dupilumab effectiveness was comparable with the clinical trial data, despite a higher proportion of patients in the real-world cohort receiving immunosuppressant therapy, which makes improvements in efficacy harder to achieve. The committee also noted that the RWE presented supported the understanding of dupilumab's long-term clinical effectiveness and informed assumptions for the economic model.ConclusionsTo date, the majority of products receiving an EAMS designation have not presented RWE at NICE reappraisal. The case of dupilumab illustrated how RWE collected through the EAMS can be used to reduce uncertainty around how clinical trial data can be translated into clinical practice. In the future, RWE may increasingly be used to help inform NICE decisions.

External validation of computed tomography decision rules for minor head injury: prospective, multicentre cohort study in the Netherlands

ObjectiveTo externally validate four commonly used rules in computed tomography (CT) for minor head injury.DesignProspective, multicentre cohort study.SettingThree university and six non-university hospitals in the Netherlands.ParticipantsConsecutive adult patients aged 16...

Ipilimumab for Previously Untreated Unresectable Malignant Melanoma: A Critique of the Evidence.

The National Institute for Health and Care Excellence (NICE) invited the manufacturer of ipilimumab (Bristol-Myers Squibb Pharmaceuticals Limited) to submit clinical and cost-effectiveness evidence for previously untreated advanced (unresectable or metastatic) melanoma as part of the Institute's Single Technology Appraisal process. The Centre for Reviews and Dissemination and Centre for Health Economics at the University of York were commissioned to act as the independent Evidence Review Group (ERG). This article presents a summary of the manufacturer's submission of ipilimumab, the ERG review and the resulting NICE guidance TA319, issued in July 2014. Ipilimumab at a recommended dose of 3 mg/kg monotherapy was previously granted marketing authorisation by the European Medicines Agency in adult patients who had received prior therapy and was recommended by NICE in guidance TA268. In October 2013, the EMA approved the extension of this indication to previously untreated advanced melanoma patients. NICE decisions are bound by the marketing authorisation; therefore, the decision problem faced by the NICE Appraisal Committee was whether ipilimumab at a recommended dose of 3 mg/kg monotherapy was effective and cost effective compared with first-line standard of care involving dacarbazine (DTIC) and vemurafenib (for BRAF V600 mutation-positive patients). The CA184-024 trial was the primary source of clinical evidence for ipilimumab. However, this was based on a dose of 10 mg/kg with concomitant DTIC. The results over a 5-year period indicated that ipilimumab 10 mg/kg plus DTIC demonstrated a significant increase in median overall survival (OS) of 2.1 months compared with DTIC plus placebo (11.2 vs. 9.1 months). The BRIM-3 trial, which was an open-label randomised controlled trial (RCT) in BRAF V600 mutation-positive patients, was the primary source of evidence for an indirect comparison with vemurafenib. The results showed that vemurafenib increased median OS by 3.6 months compared with DTIC (13.2 vs. 9.6 months). The economic evaluation compared the costs and outcomes of ipilimumab by assuming that the 3 mg/kg dosing regimen was clinically equivalent in efficacy to an ipilimumab 10 mg/kg dosing regimen plus DTIC and by using a treatment sequencing approach that incorporated second-line active therapy and third-line best supportive care (BSC). In the first appraisal meeting, the committee recommended ipilimumab only in the context of research as part of a clinical study. This was primarily based on the lack of robust evidence to support the assumption of clinical equivalence between dosages and the absence of evidence available to inform the sequential use of treatments. Following the consultation, the manufacturer submitted additional analyses and evidence to support the cost effectiveness of ipilimumab at first line. The manufacturer's response was based on concerns relating to uncertainty surrounding the relative efficacy of ipilimumab 3 mg/kg monotherapy compared with DTIC and vemurafenib, comparability of the patient populations at first and second line, and the effects of concomitant DTIC. These additional analyses indicated that the incremental cost-effectiveness ratio (ICER) was £ 47,900 per quality-adjusted life-year (QALY) gained for ipilimumab compared with DTIC and £ 28,600 per QALY gained for ipilimumab compared with vemurafenib. Following consideration of the additional evidence and the responses from a large number of consultees and commentators, the committee recommended ipilimumab as an option for adults with previously untreated advanced melanoma.

Opportunity Costs of Implementing Nice Decisions in NHS Wales.

Background: When a technology is recommended by the National Institute for Health and Care Excellence (NICE), the National Health Service (NHS) is mandated to provide the funding to accommodate it within three months of publication. Explicit in NICE’s approach to health technology assessment (HTA) is the assumption that the approval of a new, cost-increasing technology will result in the displacement of an existing, less cost-effective health care programme from elsewhere in the NHS. Notwithstanding the research which has been undertaken to establish what NICE’s cost effectiveness threshold should be, to date there has been no attempt to identify the actual opportunity costs of specific NICE decisions. Understanding what, in practice, is foregone when new cost-increasing technologies are introduced is important for an understanding of the effects of HTA.Objectives: The objectives of this paper were to document how in practice local NHS commissioners in Wales accommodated financial shocks arising from technology appraisals (TAs) issued by NICE and from other cost pressures. We also aimed to investigate how prioritisation decisions were made by budget holders in the NHS.Methods: Semi-structured interviews were conducted with Finance Directors and Medical Directors from all seven Local Health Boards (LHBs) in NHS Wales. These interviews covered prioritisation processes, as well as methods of financing NICE TAs and other financial “shocks” at each LHB. We then undertook a systematic identification of themes and topics from the information recorded. The study relates to the period October 2010 to March 2013.Results: The financial impact of NICE TAs is generally anticipated and planned for in advance and the majority of LHBs have contingency funds available to cope with these and other “shocks” within-period. Efficiency savings (defined as reductions in costs with no assumed reductions in quality) were a major source of funds for cost pressures of all kinds. Service displacements were not linkable to particular NICE TAs and there appears to be a general lack of explicit prioritisation activities. The Welsh Government has, on occasion, acted as the funder of last resort.Conclusions: Services may be displaced as part of a response to the cumulative impact of all types of cost pressures, including cost-increasing health technologies newly recommended by NICE, but such displacements were not direct responses to the publication of individual NICE TAs. The additional cost pressure represented by a new NICE TA is likely to be accommodated at least partly by greater efficiency and increased expenditure rather than displacement of services.

The Influence of Cost-Effectiveness and Other Factors on Nice Decisions.

The National Institute for Health and Care Excellence (NICE) emphasises that cost-effectiveness is not the only consideration in health technology appraisal and is increasingly explicit about other factors considered relevant but not the weight attached to each. The objective of this study is to investigate the influence of cost-effectiveness and other factors on NICE decisions and whether NICE's decision-making has changed over time. We model NICE's decisions as binary choices for or against a health care technology in a specific patient group. Independent variables comprised of the following: clinical and economic evidence; characteristics of patients, disease or treatment; and contextual factors potentially affecting decision-making. Data on all NICE decisions published by December 2011 were obtained from HTAinSite [www.htainsite.com]. Cost-effectiveness alone correctly predicted 82% of decisions; few other variables were significant and alternative model specifications had similar performance. There was no evidence that the threshold has changed significantly over time. The model with highest prediction accuracy suggested that technologies costing £40 000 per quality-adjusted life-year (QALY) have a 50% chance of NICE rejection (75% at £52 000/QALY; 25% at £27 000/QALY). Past NICE decisions appear to have been based on a higher threshold than £20 000-£30 000/QALY. However, this may reflect consideration of other factors that cannot be easily quantified. © 2014 The Authors. Health Economics published by John Wiley & Sons Ltd.

Opportunity Costs of Implementing Nice Decisions in NHS Wales

Background: When a technology is recommended by the National Institute for Health and Care Excellence (NICE), the National Health Service (NHS) is mandated to provide the funding to accommodate it within three months of publication. Explicit in NICE’s approach to health technology assessment (HTA) is the assumption that the approval of a new, cost-increasing technology will result in the displacement of an existing, less cost-effective health care programme from elsewhere in the NHS. Notwithstanding the research which has been undertaken to establish what NICE’s cost effectiveness threshold should be, to date there has been no attempt to identify the actual opportunity costs of specific NICE decisions. Understanding what, in practice, is foregone when new cost-increasing technologies are introduced is important for an understanding of the effects of HTA.Objectives: The objectives of this paper were to document how in practice local NHS commissioners in Wales accommodated financial shocks arising from technology appraisals (TAs) issued by NICE and from other cost pressures. We also aimed to investigate how prioritisation decisions were made by budget holders in the NHS.Methods: Semi-structured interviews were conducted with Finance Directors and Medical Directors from all seven Local Health Boards (LHBs) in NHS Wales. These interviews covered prioritisation processes, as well as methods of financing NICE TAs and other financial “shocks” at each LHB. We then undertook a systematic identification of themes and topics from the information recorded. The study relates to the period October 2010 to March 2013.Results: The financial impact of NICE TAs is generally anticipated and planned for in advance and the majority of LHBs have contingency funds available to cope with these and other “shocks” within-period. Efficiency savings (defined as reductions in costs with no assumed reductions in quality) were a major source of funds for cost pressures of all kinds. Service displacements were not linkable to particular NICE TAs and there appears to be a general lack of explicit prioritisation activities. The Welsh Government has, on occasion, acted as the funder of last resort.Conclusions: Services may be displaced as part of a response to the cumulative impact of all types of cost pressures, including cost-increasing health technologies newly recommended by NICE, but such displacements were not direct responses to the publication of individual NICE TAs. The additional cost pressure represented by a new NICE TA is likely to be accommodated at least partly by greater efficiency and increased expenditure rather than displacement of services.

Health technology assessments in England: an analysis of the NICE Medical Technologies Evaluation Programme

Many countries undertaking health technology assessments (HTAs) to inform decisions on the reimbursement of technologies focus solely on the analysis of pharmaceuticals [1, 2]. In the UK, the responsibility for undertaking HTAs falls mainly to the National Institute for Health and Care Excellence (NICE). NICE is an independent organisation whose role is to provide guidance on health technologies and clinical practice for the National Health Service (NHS) in England and Wales [3], and it is regarded as a world leader in the HTA field. In contrast to HTA bodies in most other countries, NICE has been given a wider remit to assess the full range of technologies (i.e. not just pharmaceuticals) [4]. There are difficulties present when assessing non-pharmaceutical health technologies, which can reduce the applicability of methods routinely used in the assessment of pharmaceuticals. Nevertheless, it is important not to undertake HTAs only on those technologies where the assessment process is straightforward, otherwise the allocation of resources between the different types of technology may be distorted by an imbalance in the information supporting decisions [2]. In order to overcome the aforementioned difficulties and ensure that the assessments of all forms of health technology are useful in decision-making, NICE has developed four appraisal programmes that are each overseen by a separate committee (technology appraisals, medical technologies, diagnostics and public health). At this point a distinction needs to be made between two sections of the NICE decision process: assessment and appraisal. An assessment is the review and analysis of data and other evidence, whereas appraisal is the a priori consideration of the evidence on benefits and costs of different courses of action in order to distinguish which is superior. The assessment of technologies is undertaken by the NICE technical team and an independent review group, with the manufacturer of the technology also having a major role. The evidence generated from this process is then passed onto one of the NICE committees, who have to make a decision regarding the suitability of the technology for the NHS, the appraisal section of the process. There are four NICE committees, each of which is responsible for one of the four programmes discussed previously. The newest of them is the Medical Technologies Advisory Committee (MTAC). The MTAC assesses a specific subset of health technologies, which can loosely be defined as medical devices. A full description of the technologies that are assessed by the MTAC can be found in the Medical Technologies Evaluation Programme (MTEP) methods guide [5], referred to as ‘MTEP guidance’ in the remainder of this paper. This methods guide stipulates the methodology that manufacturers must follow when undertaking economic evaluations of medical devices that are to be assessed by the MTAC. NICE aims for consistency across its four committees. However, a comparison of the methodology outlined in the MTEP guidance with those used by the other NICE decision-making committees shows some significant differences. The possible W. Green (&) York Health Economics Consortium, University of York, Market Square, Heslington, York YO10 5NH, UK e-mail: william.green@york.ac.uk

The Influence of Cost-Effectiveness and Other Factors on NICE Decisions

Background: The National Institute for Health and Care Excellence (NICE) emphasises that cost-effectiveness is not the only consideration in health technology appraisal and is increasingly explicit about other factors considered relevant. Observing NICE decisions and the evidence considered in each appraisal allows us to “reveal” its implicit weights. Objectives: This study aims to investigate the influence of cost-effectiveness and other factors on NICE decisions and to investigate whether NICE’s decision-making has changed through time. Methods: We build on and extend the modelling approaches in Devlin and Parkin (2004) and Dakin et al (2006). Wemodel NICE’s decisions as binary choices: i.e. recommendations for or against use of a health care technology in a specific patient group. Independent variables comprised: the clinical and economic evidence regarding the technology; the characteristics of the patients, disease or treatment; and contextual factors affecting the conduct of health technology appraisal. Data on all NICE decisions published by December 2011 were obtained from HTAinSite.Results: Cost-effectiveness alone correctly predicted 82% of decisions; few other variables were significant and alternative model specifications led to very small variations in model performance. The odds of a positive NICE recommendation differed significantly between musculoskeletal disease, respiratory disease, cancer and other conditions. The accuracy with which the model predicted NICE recommendations was slightly improved by allowing for end of life criteria, uncertainty, publication date, clinical evidence, only treatment, paediatric population, patient group evidence, appraisal process, orphan status, innovation and use of probabilistic sensitivity analysis, although these variables were not statistically significant. Although there was a non-significant trend towards more recent decisions having a higher chance of a positive recommendation, there is currently no evidence that the threshold has changed over time. The model with highest prediction accuracy suggested that a technology costing £40,000/quality-adjusted life year (QALY) would have a 50% chance of NICE rejection (75% at £52,000/QALY; 25% at £27,000/QALY). Discussion: Past NICE decisions appear to have been based on a higher threshold than the £20,000-£30,000/QALY range that is explicitly stated. However, this finding may reflect consideration of other factors that drive a small number of NICE decisions or cannot be easily quantified.